uniQure downgraded after FDA backs sham-controlled trial for Huntington’s therapy

Investing.com -- Analysts downgraded uniQure N.V. after the U.S. Food and Drug Administration signalled it would require a sham surgery-controlled Phase 3 trial for the company’s Huntington’s disease therapy, dimming hopes for a faster approval path.

A sham-controlled trial is a type of randomized clinical trial where a control group receives an inactive, simulated procedure (the "sham") designed to mimic an active, often surgical or device-based, intervention

Mizuho downgraded the stock to Neutral from Outperform and slashed its price target to $12 from $33.

The brokerage said confidence in regulatory flexibility for AMT-130 fell materially following minutes from a Type A meeting with the FDA.

While Mizuho had considered the possibility of a Phase 3 trial, it had expected a more flexible approach, potentially using historical controls.

Instead, the FDA “strongly” recommended a prospective sham-controlled study. Mizuho said such a trial would be difficult to execute and could raise ethical concerns.

The firm pushed its expected launch for AMT-130 to 2030 from 2028 and cut its probability of success to 15% from 65%, though it kept its unadjusted sales forecast unchanged. Its $12 target reflects a risk-adjusted valuation of $6 for AMT-130 and $6 for estimated 2026 year-end cash.

BofA also downgraded the shares to Sector Perform and lowered its target price to $11, calling the update disappointing.

The bank said the FDA is unwilling to approve AMT-130 based on comparisons to natural history data and instead favors a randomized, double-blind, sham-controlled study. A Type B meeting to discuss trial design is expected in the second quarter of 2026.

Both firms said ongoing Phase 1/2 data, including a four-year readout expected in the third quarter of 2026, are unlikely to support approval under the current regulatory stance.

BofA added that the broader pipeline remains early. Fabry disease data were positive, but mid- and high-dose cohorts are on pause. The SOD1-ALS study remains on hold after a serious adverse event, while mesial temporal lobe epilepsy data remain limited, with an update expected in the second quarter of 2026.

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